Canavan Disease Research Paper

There are two main groups of gene therapy and they are germ line gene therapy and somatic gene therapy (Baksh, 2007). Germ line therapy consists of germ cells being modified by the introduction of functional genes, which are ordinarily integrated into their genomes (Garbutt et al., 2011). DNA encodes the therapeutic gene and replaces the mutated gene so the new gene can treat the disease. Germ line

According to the National Hemophilia Foundation (.n d.), people with VWD experience recurrent nosebleeds, easy bruising and extreme bleeding for the duration of and after invasive procedures, such as tooth removals and surgery. Women often experience menorrhagia, heavy menstrual periods that last lengthier than usual, and hemorrhaging after giving birth. The signs and symptoms depends on the severity of the disease.

Gene therapy is an experimental technique that is used to prevent disease. For future reference, this technique may treat disorders. It may add or remove genes to get rid of a genetic disorder. Gene therapy is a heavily debated subject. There are very respectful points on both sides of this debated topic. The pro side believes that gene therapy is the best thing that is happening to our society. However, opposers of gene therapy believe that people should refrain from proceeding with this biological advancement and it is ethnically incorrect.

Batten disease is also a fatal hereditary disorder of the central nervous system occurring in childhood. The symptoms include formation of lipopigments in body tissues and early symptoms include personality and behavior changes, clumsiness or stumbling. As the disease advances children may experience mental impairment, loss of sight and motor skills, become blind and bedridden. Lafora body disease is a rare genetic disorder responsible for causing progressive dementia and movement problems. The symptoms arise in the early childhood or late teens and are characterized by the presence of Lafora bodies in brain, skin, muscles and liver and death of child within 2-10 years.

Gene therapy is the insertion of normal or genetically altered genes into cells to replace defective genes. The gene is usually inserted into

The modified cells would then continue to replicate as normal cells. The in vivo approach involves the gene being directly injected into the affected tissues or into the bloodstream(Health Canada, 2012).. Although both these approaches involve the gene entering a patient’s cell in one way or another, the gene itself cannot just be entered into a cell. To be able to enter a cell the gene must use a carrier known as a vector in order for the gene to effectively enter the cell and start producing the correct proteins. A vector is a type of virus (usually adenoviruses, retroviruses and herpes simplex virus) where the virus’ DNA is removed and replaced with the gene that is required(U.S National Library of Medicine,2018). The vector can then either be applied to body cells which is called somatic gene therapy, or more controversially the gametes of the patient which is called germline gene therapy(legal in the US but banned in Canada under Section 5 of the Assisted Human Reproduction Act and other countries like Germany, Australia and UK) (Legislative Services Branch,2004). Both types of gene therapy will cause the vector to enter the

Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional

The Canavans’ disease is a rare and fatal degenerative brain disease caused by a defective gene that impairs the formation of myelin in the brain (Purnell, 2013). At the mid-infancy, the signs and symptoms starts include, developmental delay, loss of vision and loss of reflexes and death at the age of ten (Purnell, 2013).

Gene therapy is an experimental treatment, which is used to replace mutated, improperly functioning genes with regular DNA. There are several gene therapy methods, most of which involve using a vector to transport the corrected gene into targeted cells. Retro viruses, adeno viruses, adeno ascosciated viruses and liposomes are commonly used as vectors. Naked DNA (DNA without

Gene therapy is a rapidly developing technology first used in 1990.1 It involves the use of modified DNA inserted into an organism with a missing or mutated sequence. There are two methods of delivering this correction into the patient’s cells. In the viral method, a virus containing the vector with the corrected form of the gene, infects the cell naturally. Adenoviruses, adeno-associated viruses, lentiviruses, and retroviruses can all be used. They each utilize a different method of delivery and form of DNA. Adeno-associated viruses and lentiviruses are the easiest forms to construct.2 The corrected DNA faces many obstacles to gene expression and ultimately protein production. The viruses must target and enter the correct cell, avoid

Gene Therapy is an experimental form of treatment that aims to get rid of genetic conditions at their source. It also may be used to replace a faulty gene with a working version or introduce a new gene to cure a condition or modify its effects. ( Victoria State Government, 2011) It is designed to introduce genetic material into cells to compensate for abnormal genes or to create a beneficial protein within the gene. If a mutated or incomplete gene creates a protein needed that is faulty or not present, gene therapy has the ability to introduce a copy of that specific gene to restore the function of the protein within that gene.

A new form of medicine with the potential to save and improve millions of live is gene therapy, or gene replacement therapy. Gene therapy is the use of DNA as a pharmaceutical agent used to treat or cure diseases or disorders caused by defective or missing genes. There are three methods of gene therapy: replacing a mutated gene with a healthy copy of the same gene, inactivating a gene that is not functioning properly, or introducing a completely new gene to help the patient fight a disease. Gene therapy is a promising treatment option, however, the technique has yet to be perfected and is still dangerous and experimental.

According to Blau and Springer (1995), gene therapy is a newly rising approach at the end of the 20th century and aims to open new door for the treatment of various human diseases based on transfer of genetic materials to cells. The recent gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy is not inherited and thus it cannot be passed down on to a person’s children in the future. Meanwhile, gene therapy could also be targeted to egg and sperm cells (knowns as germline gene therapy) which would allow the inserted gene to be passed on to the progeny. Germline gene therapy allows the correction of disease-causing

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. ("Gene Therapy," n.d.). In the United States, gene therapy is only available in a research setting.

If a treatment is implicated before the child is born – germline gene therapy – he or she could lose all trace of that defective gene, and therefore wouldn’t pass the disease on to future generations. In somatic gene therapy, treatment is conducted when the patient is an